Bionic Sight, a technology company that develops optogenetic gene therapy vectors and devices to treat retinal degenerative diseases, said it is one step closer to “cracking the code” for blindness following a successful clinical trial of its BS01 gene therapy to treat retinitis pigmentosa (RP).
Bionic Sight’s approach involves two components: an optogenetic treatment, currently on trial, which bestows light sensitivity to retinal ganglion cells after the eye’s photoreceptors are lost due to advanced retinal diseases, such as RP; and a device, worn like glasses, which sends a vision-enabling code through the ganglion cells and optic nerve to the brain.
Unlike other emerging optogenetic approaches, which may use glasses or goggles to enhance the shape and intensity of the image, the Bionic Sight device produces electrical impulses, similar to those produced by photoreceptors, said Bionic Sight founder Dr Sheila Nirenberg, professor of physiology and biophysics at Weill Medical College of Cornell University.
“A key issue for producing meaningful vision is to send signals to the brain that the brain can understand. When the optic nerve communicates with the brain, it does it in a code. The code is in the form of patterns of neural pulses that tell the brain what you’re looking at – faces, objects, a clear path ahead of you. If you can deliver signals in the language the brain understands, then meaningful perceptions become possible.”
The open-label phase I/II trial is a dose escalation study in patients diagnosed with advanced stage RP. Participants are enrolled in four groups with the first three receiving a low, middle and high dose, while the fourth group will receive the maximum tolerated dose as determined by the first three groups.
